SNN’s Robert Kraft spoke with Jeffrey Bacha, President and CEO of DelMar Pharmaceuticals about the company’s drug candidates, what orphan cancer indications DelMar is targeting, and 2015 objectives.

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Thursday, February 26, 2015

DelMar Pharmaceuticals, Inc. (OTCQX: DMPI) is developing drug candidates targeting orphan cancer indications. The comapany’s lead product candidate, VAL-083, which is approved as a cancer chemotherapeutic in China for the treatment of chronic myelogenous leukemia ("CML") and lung cancer; VAL-083 has not been approved for any indications outside of China, according to the company’s website.

1. Mr. Bacha, let’s start off with an overview of DelMar Pharmaceuticals, Inc.

We founded DelMar Pharmaceuticals in 2010 to focus on developing cancer therapies for patients who were failing modern biologic or targeted therapies. We took the company public in 2013. DelMar is traded on the OTCQX under the symbol “DMPI”.

Our business strategy is to take “proven” cancer therapies, where there is evidence of clinical activity against one or more types of cancer and, through DelMar’s research, develop modern biological data to better understand the way the drug attacks cancer. We then consider that “mechanism of action” in the context of historical clinical activity – “the modern understanding of how the drug works combined with where we know it worked historically” – to see if we might be able to solve an unmet medical need in the treatment of cancer today.

If the answer is “yes”, we establish new intellectual property through patent filings and seek to advance toward FDA approval in the most streamlined manner possible.

We’ve actually identified a number drug candidates meeting these criteria that were originally studied at the National Cancer Institutes in the United States, but were never commercialized for one reason or another.

The DelMar clinical development team has implemented this strategy successfully before – leading to successful FDA approval of another drug candidate at a company that was eventually acquired. When that company was acquired, key members of the team reformed and established DelMar Pharmaceuticals. DelMar’s clinical operations facilities in Menlo Park, CA are actually the same address! So, this is a proven business model being implemented by a team with a history of success.

2. According to the company’s website, DMPI’s lead drug candidate is VAL-083, which is currently in Phase II clinical trials – what indications are you currently working on in this clinical trial, and how is it progressing? Where are you conducting the clinical trials?

Our lead indication with VAL-083 is glioblastoma multiforme, or GBM as it is sometimes known. GBM is the most common and aggressive form of brain cancer – affecting about 15,000 new patients per year in the United States alone.

Unfortunately, most GBM patients fail to respond to treatment and there is little that can be done for them. The average survival from the time of diagnosis, even with modern cancer care, is less than two years.

VAL-083 is a cancer chemotherapy that was investigated by the National Cancer Institutes in the United States in the “original” war on cancer during the late 1970s and early 80s. In previous NCI-sponsored human clinical trials, VAL-083 was shown to be active against a number of different cancers, including GBM.

What we’ve done is to develop a new understanding of how VAL-083 attacks cancer and shown that this unique anti-tumor mechanism is different than other chemotherapies used to treat GBM. Our data also shows that, because of this unique mechanism, VAL-083 is active where other GBM chemotherapies fail.

DelMar Pharmaceuticals initiated new clinical trials in the United States with VAL-083 as a potential new treatment for refractory GBM – targeting patients who have failed other available therapies. Our clinical trial is currently being conducted at three sites in the United States: UC San Francisco and two sites affiliated with the Sarah Cannon Cancer Research Center – one in Nashville and one in Sarasota, FL.

The goal of our initial Phase I/II clinical trial is to define the maximum tolerated dose, or MTD, in GBM patients who have been treated with and failed radiation therapy, a chemotherapy called Temodar®, and another drug called Avastin® – more than half of the patients diagnosed with GBM fail these therapies and have no viable treatment options.

Establishing the MTD in these patients will allow us to get as much VAL-083 chemotherapy to the tumor as possible while managing the side-effects associated with the drug. Our goal is to “hit the tumor harder, more often” compared to previous GBM clinical trials conducted by the NCI. So far, we’ve shown that our modernized dosing regimen can deliver at least twice as much VAL-083 safely, so we’re achieving our aims in this regard!

Once MTD is confirmed, we’ll advance VAL-083 into a Phase II/III registration directed trial in refractory GBM patients. We anticipate that the Phase II/III registration directed trial will involve about 100 GBM patients who have failed other therapies.

We’ll also be able to leverage our modernized dosing regimen to initiate additional clinical trials in newly diagnosed GBM patients whose tumors have characteristics known to cause resistance to other chemotherapies used in the treatment of GBM.

Based on VAL-083’s unique mechanism of action, we believe DelMar is positioned to address huge unmet medical needs in the treatment of GBM.

We’re also planning to explore VAL-083 in other cancers where its unique mechanism supports a potential solution to unmet medical needs. Our next focus will be in non-small cell lung cancer, which represents the highest mortality rate of all cancers. VAL-083 is currently approved for lung cancer in China, but not widely used. Our data suggests that we can reposition the drug in that market, and also meet patent needs in lung cancer around the world.

3. What is the market for treatment of glioblastoma multiforme (GBM)? Who is affected by this rare disease?

The current front-line therapy for GBM, Temodar®, achieved annual sales of nearly $1 billion prior to coming off patent in 2013.

GBM affects approximately 3 of every 100,000 people world-wide. This translates to approximately 15,000 new patients every year in the United States.

So, compared to some other cancers, it would be considered “rare”; however, because GBM is so deadly in nature and generally affects people in the prime of life, it is well known to the public. It is the cancer that Sen. Ted Kennedy died from, for example.

There doesn’t appear to be any connection to race or gender … GBM is an equal opportunity killer.

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4. On February 2, 2012, DelMar was granted U.S. Orphan Drug Designation for VAL-083 for the treatment of GBM. For our audience, what does U.S. orphan designation mean and how is one granted U.S. orphan designation for a drug candidate? How does receiving orphan drug designation affect time-to-market?

VAL-083 has been granted orphan drug protection for the treatment of glioma – brain cancers arising from glial cells, which includes GBM – by the US FDA and the European Medicines Authority.

The Orphan Drug Designation in the United States is available for drugs being developed to treat a disease with a prevalence of less than 250,000 patients in the United States. Gliomas, including GBM, meet this criterion.

A product can receive an orphan drug designation following application to the FDA Office of Orphan Products Development, or OOPD. The OOPD has a mission to advance the evaluation and development of products for the diagnosis and treatment of orphan diseases. Applications are evaluated on scientific merit as well as to confirm that the target disease fits the definition of a rare or “orphan” disease.

Drugs granted orphan drug protection generally follow the same regulatory development path as any other pharmaceutical product. However, incentives such as scientific advice and reduction or waiver of registration fees and access to specialized grant funding are available to support and accelerate development of orphan drug candidates.

That said, because of the small number of patients with an orphan disease, the size of clinical trials required for approval are often relatively small. For example, Avastin® the last drug approved in the treatment of GBM was approved on an 85 patient open label study.

In addition, drugs with an Orphan Drug Designation are granted “market exclusivity” following approval. This means that only first company receiving approval can market a product with the same active ingredient for a particular orphan disease. The period of exclusivity is seven years in the United States and ten years in Canada and Europe.

This provides DelMar with market exclusivity for VAL-083 in gliomas upon approval. In addition, we have filed several new patents. To date, three US patents and one international patent have been allowed, so we have patents that will protect VAL-083 well beyond the expiry of the orphan drug exclusivity period.

The FDA can also grant “accelerated approval” and “fast-track” regulatory pathways for drug candidates targeting diseases where there is no available therapy, such as GBM. Accelerated approval allows approval based on a surrogate endpoint, such as radiographic imaging and fast-track allows for a “rolling-review” of a new drug application so that clinical data can be submitted to the FDA almost in real time rather than waiting for the end of a clinical trial. Both of these pathways were established by the FDA enabled the faster approval of new drugs to meet significant unmet medical needs.

GBM certainly falls within the definition of a significant unmet medical need and DelMar will seek to take advantage of these programs to accelerate the approval process wherever possible.

It is important for us to make VAL-083 available to doctors and patients as soon as we can do so.

5. How is U.S. orphan designation different from receiving orphan designation in other parts of the world? Is there a difference? If so, how?

An orphan drug designation is available from a number of countries and regions around the world, including Europe, the United States, Canada, Japan, Australia, and several countries in Asia have similar legislation approved or under consideration.

The criteria and processes for designation are not internationally harmonized. For example, in the United States, the primary evaluation is based on prevalence of disease, but in Europe an application not only has to be supported by disease prevalence, but data demonstrating the potential to address a specific unmet need.

The availability of market exclusivity also varies – seven years in the US and 10 years in the EU – as does the availability of direct government payment for therapy.

But while process and scope may differ from country to country, the general premise is the same: To provide incentives for development of new diagnostics and treatments to meet the unmet medical needs of patients suffering from rare diseases as quickly as possible.

6. What is your background?

I’ve been involved in pharmaceutical company building for over 20 years.

I have a degree in BioPhysics from the University of California, San Diego and an MBA with a focus in finance from the Goizueta Business School at Emory University.

I spent some time at the research benchtop at a private diagnostics company in San Diego before deciding to pursue an MBA. After business school I spent several years with KPMG Health Ventures– a consulting group within the accounting firm that works with small and mid-size healthcare and pharmaceutical companies to support clients’ strategic and financing initiatives. I was eventually recruited by a group of investors as part of a management restructuring at a specialty pharmaceutical company. I’ve also been involved in launching new venture-capital backed start up from the University of British Columbia and have consulted with several small and mid-stage companies prior to co-founding DelMar Pharmaceuticals in 2010.

7. How does 2015 look for DelMar Pharmaceuticals?

We’re excited about a big year ahead for DelMar Pharmaceuticals.

We expect to define the MTD from our Phase I/II clinical trial with VAL-083 in refractory GBM in the coming months. This will position us to rapidly advance VAL-083 into late-stage Phase II/III clinical trials in refractory GBM and to expand the our development to include newly diagnosed GBM patients whose tumors exhibit molecular features known to cause resistance to other chemotherapies used in the treatment of brain cancer.

We also expect to initiate new clinical work in non-small cell lung cancer during the first half of 2015. This is a great opportunity for us because our manufacturing partner, Guangxi Wuzhou Pharmaceuticals, – the company holding the approval in China – has agreed to fund the currently planned clinical trials in lung cancer.

We’re continuing to build our intellectual property portfolio through new patent filings and are in the process of planning to “up-list” our stock to a national stock exchange in the United States such as NASDAQ or NYSE-MKT.

So, 2015 should be a big year for DelMar Pharmaceuticals. As we go forward we anticipate presenting data from our research and clinical development programs at peer reviewed cancer meetings such as the American Association of Cancer Research (AACR), the American Society of Clinical Oncology (ASCO) and the Society for Neuro-Oncology (SNO) annual meetings. We invite you to watch our progress as we advance VAL-083 as a potential new therapy for the treatment of orphan and other cancers with significant unmet medical needs.

For more information about DelMar Pharmaceuticals, Inc., check out their website: www.DelMarPharma.com